Lumos Pharma Inc.’s novel approach with LUM-201 in pediatric growth hormone deficiency paid off in top-line results, and the company is laying the groundwork for a phase III study next year. Pisit “Duke” Pitukcheewanont, senior vice president of global clinical development and medical affairs for Lumos, told BioWorld that the ability of LUM-201 to generate hormone at the normal range, promoting growth comparable to standard-of-care daily or weekly injections is “groundbreaking.”
While executives of Durect Corp. plugged “compelling” reduction in mortality from a phase IIb study testing DNMT inhibitor larsucosterol in severe alcohol-associated hepatitis and looked ahead to a potential registrational trial, investors focused on the fact that the study, called Ahfirm, fell short of statistical significance on both primary and secondary endpoints, sending shares (NASDAQ:DRRX) down 79%.
Otsuka Pharmaceutical Co. Ltd.’s sibeprenlimab (VIS-649) met the primary efficacy endpoint in a phase II trial for treating immunoglobulin A nephropathy (IgAN), demonstrating that 12 months of treatment resulted in significant reductions in proteinuria compared to placebo.
Paxmedica Inc.’s shares (NASDAQ:PXMD) surged by 179% in early trading Nov. 7 after reporting the publication of phase II autism spectrum disorder results for PAX-101 (suramin) intravenous infusions. Suramin, an anti-trypanosomal and anti-purinergic agent introduced in 1923 for East African sleeping sickness, showed a statistically significant improvement at the 10-mg/kg dose in a secondary endpoint of Clinical Global Impressions – Improvement scale vs. placebo.
Having rolled out less-than-stellar phase II data with oral TYK2 inhibitor VTX-958 in moderate to severe plaque psoriasis, Ventyx Biosciences Inc. faces Wall Street speculation regarding the odds for the ongoing effort in Crohn’s disease (CD), due for an interim analysis in the first quarter of next year.
Top-line results from Moonlake Immunotherapeutics AG’s phase II study of sonelokimab treating active psoriatic arthritis encouraged the company but discouraged investors.
Among the avalanche of abstracts for the upcoming American Society of Hematology (ASH) annual conference released early on Nov. 2 were data from Arcellx Inc.’s phase I study of CART-ddBCMA in patients with relapsed or refractory multiple myeloma. The results caught investors’ attention as the company’s stock (NASDAQ:ACLX) jumped 18.5% upward to close at $40.77 per share.
Oncolys Biopharma Inc.’s lead compound Telomelysin (suratadenoturev, OBP-301) met the primary endpoint of exceeding the predefined threshold of local complete response, confirming clinical benefit in a phase II study in locally advanced esophageal cancer combined with radiation.
With Deciphera Pharmaceuticals Inc.’s positive top-line data in hand from the pivotal phase III study called Motion, Wall Street speculation turned to the comparative safety and regulatory odds for vimseltinib (which analysts often refer to as “vim”) in patients with tenosynovial giant cell tumor (TGCT) not amenable to surgery.
Clinical trial data from January-September 2023 was up 1.79% compared to the same time period last year. In the first three quarters of this year, BioWorld reported on 2,611 drugs in phase I-III, compared to 2,565 in Q1-Q3 2022. The number of trial updates is down 13.74% from 3,027 in 2021 and also down from the 2,738 in 2020, but up from 2,251 updates noted in 2019.
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